Clinical Trials 101
Clinical trials are research studies conducted in people to assess whether treatments are effective and safe. Clinical research is the only way to turn promising science into treatments for people. Without clinical research and human volunteers, we cannot create better treatment and prevention strategies for the disease.
Clinical trials on ALD/AMN are designed to test new ways to:
- Find and diagnose ALD/AMN
- Treat ALD/AMN
- Prevent ALD/AMN
- Manage symptoms of ALD/AMN and side effects from treatment
Phases of drug clinical trials
Laboratory-based research, or pre-clinical research, takes place before any testing in humans is performed. These studies provide a scientific basis for a potential drug or treatment that may be safe and effective on people. Once a drug has been developed using preclinical research, the drug must complete a three-phase clinical trial process (Phase I, II, and III) before the FDA can approve the drug for public’s use. The drugs must perform well in each phase to progress to the next one. It often takes 10 to 15 years or more to complete all 3 phases of clinical trials.
- Phase I: This is the first stage of human testing. It typically involves fewer than 20 healthy volunteers or very sick patients for whom treatment options are lacking. The goal of this phase is to understand the effect the drug has on human subjects and to examine potential risks and side effects of a drug. During this phase, researchers also determine how the drug should be used or delivered.
- Phase II: Hundreds of volunteers who have the condition the drug is designed to treat are enrolled to Phase II. This phase provides more information about drug safety and helps determine dosage of a drug. In some instances, multiple Phase II trials are conducted to test the compound in a variety of patient populations or indications. This phase can last several years.
- Phase III: Several hundred to thousands of volunteers who have the condition participate in multiple study sites worldwide. This phase compares the drug or vaccine to a placebo or standard intervention, to evaluate safety and effectiveness. The FDA will consider the results of all three phases of clinical trials when determining whether to approve the new drug for the public.
- Phase IV: This often occurs after the FDA approves the new drug. During this phase, researchers continue to monitor the health of people taking the medication to gain further insight into its long-term safety and effectiveness.
Most ALD/AMN patients and caregivers want to do everything they can to help find new treatments for the disease. The idea of trying an experimental therapy is often regarded positively, despite knowing that risks may be present. However, some people have misgivings when they learn that some participants in a trial may receive a placebo rather than the drug being tested.
Why is clinical research so important to finding a cure?
People with ALD who enroll in a clinical trial are contributing to improved health care for everyone with the disease. Even when the results of a trial are negative, we learn more about the disease as well as how to look for more promising new treatments. The more researchers and the public know about ALD, the greater the chance of finding a treatment that prevent or reverses the symptoms of ALD.
Because ALD is a rare hereditary disease, it is very important to enroll people who have the disease as well as family members who may be carriers for the disease. Recruiting and retaining trial participants is crucial to better understand the long-term effects of a treatment on disease symptoms and outcome. When you take part in a clinical trial, you add to the knowledge about ALD and help to accelerate the process to find potential intervention and preventative strategies.
What should people consider before participating in a trial?
People should know as much as possible about the clinical trial and feel comfortable asking the members of the health care team questions about it, the care expected while in a trial, and the cost of the trial. The following questions might be helpful for the participant to discuss with the health care team. Some of the answers to these questions are found in the informed consent document.
- What is the purpose of the study?
- Who is going to be in the study?
- Why do researchers believe the new treatment being tested may be effective? Has it been tested before?
- What kinds of tests and treatments are involved?
- How do the possible risks, side effects, and benefits in the study compare with my current treatment?
- How might this trial affect my daily life?
- How long will the trial last?
- Will hospitalization be required?
- Who will pay for the treatment?
- Will I be reimbursed for other expenses?
- What type of long-term follow up care is part of this study?
- How will I know that the treatment is working?
- Will results of the trials be provided to me?
- Who will be in charge of my care?
- Will the information gathered in the study be shared to others in the research community like ALD Connect?
We recommend visiting the Center for Information and Study on Clinical Research Participation (CISCRP) webpage for additional educational material on clinical trials. CISCRP is an independent non-profit organization dedicated to educating and informing the public and patients about clinical research. CISCRP also provides information and resources to help research and health professionals better serve their patients and study volunteers. CISCRP has excellent educational videos on their website which illustrate some best practices for clinical trial participants. Click here to see the videos.